- Upregulation of SCN1A gene expression by targeting regulatory RNAs
- Company to advance oligonucleotide development candidate for Dravet Syndrome; expected to file IND by YE 2022
CAMBRIDGE, Mass., December 3, 2021 – CAMP4 Therapeutics, a biotechnology company harnessing the power of RNA to restore healthy gene expression, today announced the presentation of in vivo proof-of-concept data demonstrating the potential therapeutic utility of its lead oligonucleotide candidate for the treatment of Dravet Syndrome, a form of severe genetic epilepsy. The data, which show increases in SCN1A gene expression in non-human primates and reductions in seizures in mouse disease models, will be presented at the American Epilepsy Society (AES) Annual Meeting being held virtually and in person in Chicago, IL from December 3-7, 2021.
Loss of function of the SCN1A gene leads to disease in the majority of Dravet Syndrome patients. CAMP4’s candidate molecule, CMP-SCN, is designed to directly upregulate endogenous gene expression of SCN1A by targeting Natural Antisense Transcripts (NAT), a subset of regulatory RNAs (“regRNAs”) that control mRNA transcription. Importantly, CMP-SCN targets the SCN1A-NAT that is expressed stably from birth to adulthood in order to achieve a consistent therapeutic effect irrespective of patient’s age.
“These data collectively validate our disease-modifying strategy for Dravet, which, we believe, will be therapeutically impactful,” said David Bumcrot, Ph.D., Chief Scientific Officer of CAMP4. “Our selection of the specific regRNA target and precise ASO design are intended to enable the development of a potent, programmable and durable therapy for this devastating early-childhood epilepsy and we now have the evidence supporting this approach and facilitating progression to IND-enabling studies.”
RegRNAs act as gene-specific rheostats that can finely modulate gene expression upstream of mRNA transcription. CAMP4 applies its RNA Actuating Platform™ comprised of extensive proprietary epigenomic and transcriptomic data to map the regulatory genome and pinpoint regRNAs and the genes they control. Oligonucleotide drug candidates, or RNA ActuatorsTM, are then screened and designed to precisely and potently engage gene-specific regRNA targets, but not to excessive levels that might generate toxicity. This approach is applicable to any genetic disease whereby even small increases in gene output can lead to meaningful therapeutic outcomes.
“The data at AES show that CMP-SCN resulted in increased SCN1A mRNA expression both in vitro and in vivo.1 In a mouse disease model, treatment with a SCN1A NAT-targeting ASO led to an increase in SCN1A expression by 25%, which was accompanied by an approximately 70% decrease in the number, frequency, amplitude and duration of seizures.1 In CAMP4’s recent non-human primate study, a low dose of CMP- SCN increased SCN1A protein expression 1.5-2 fold across multiple regions of the brain. Based on these data, CAMP4 will pursue clinical development for CMP-SCN with the expectation of filing an Investigational New Drug Application with the U.S. Food and Drug Administration by the end of 2022.”
1 Hsiao et al. eBiomedicine. 2016
The presentation can be viewed on the Publications section of the CAMP4 website.
About CAMP4 Therapeutics
At CAMP4, we are pioneering a novel approach to programmable therapeutics. We combine a deep understanding of regulatory RNA and gene expression with a complementary and customizable oligonucleotide modality. Our RNA Actuating Platform’s proprietary insights enable us to harness the power of RNA to upregulate the expression of genes and unlock the potential to create treatments for hundreds of diseases affecting millions of patients. Learn more about us at www.CAMP4tx.com and follow us @CAMP4tx.